EP 3965806 A1 20220316 - POLYNUCLEOTIDES ENCODING METHYLMALONYL-COA MUTASE FOR THE TREATMENT OF METHYLMALONIC ACIDEMIA
Title (en)
POLYNUCLEOTIDES ENCODING METHYLMALONYL-COA MUTASE FOR THE TREATMENT OF METHYLMALONIC ACIDEMIA
Title (de)
FÜR METHYLMALONYL-COA-MUTASE KODIERENDE POLYNUKLEOTIDE ZUR BEHANDLUNG VON METHYLMALONAZIDÄMIE
Title (fr)
POLYNUCLÉOTIDES CODANT POUR LA MÉTHYLMALONYL-COA MUTASE POUR LE TRAITEMENT DE L'ACIDÉMIE MÉTHYLMALONIQUE
Publication
Application
Priority
- US 201962845250 P 20190508
- US 2020032055 W 20200508
Abstract (en)
[origin: WO2020227615A1] This disclosure relates to mRNA therapy for the treatment of methylmalonic acidemia (MMA). mRNAs for use in the invention, when administered in vivo, encode methylmalonyl-CoA mutase (MUT). mRNA therapies of the disclosure increase and/or restore deficient levels of MUT expression and/or activity in subjects.
IPC 8 full level
A61K 38/52 (2006.01); C12N 9/90 (2006.01)
CPC (source: EP US)
A61K 9/0019 (2013.01 - EP US); A61K 9/1272 (2013.01 - EP US); A61K 9/5123 (2013.01 - EP US); A61K 31/7115 (2013.01 - EP US); A61K 31/713 (2013.01 - EP); A61K 48/0041 (2013.01 - EP); A61K 48/005 (2013.01 - EP); A61P 3/00 (2017.12 - EP US); C12N 9/90 (2013.01 - EP); C12Y 504/99002 (2013.01 - EP)
Citation (search report)
See references of WO 2020227615A1
Designated contracting state (EPC)
AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR
Designated extension state (EPC)
BA ME
DOCDB simple family (publication)
WO 2020227615 A1 20201112; EP 3965806 A1 20220316; MA 55899 A 20220316; US 2022370354 A1 20221124
DOCDB simple family (application)
US 2020032055 W 20200508; EP 20731624 A 20200508; MA 55899 A 20200508; US 202017608691 A 20200508